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A Study in Adults and Adolescents With Angelman Syndrome

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ClinicalTrials.gov Identifier: NCT02996305
Recruitment Status : Recruiting
First Posted : December 19, 2016
Last Update Posted : November 30, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.

Condition or disease Intervention/treatment Phase
Angelman Syndrome Drug: OV101 Regimen 1 Drug: OV101 regimen 2 Other: Placebo Phase 2

Detailed Description:
Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 75 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol
Study Start Date : January 2016
Estimated Primary Completion Date : October 2018
Estimated Study Completion Date : November 2018

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: OV101 regimen 1
OV101 once daily
Drug: OV101 Regimen 1
Other Name: Gaboxadol
Experimental: OV101 regimen 2
OV101 twice daily
Drug: OV101 regimen 2
Other Name: Gaboxadol
Placebo Comparator: Placebo
Twice daily
Other: Placebo


Outcome Measures

Primary Outcome Measures :
  1. Incidence of adverse events in placebo and active treatment groups [ Time Frame: Change from baseline to week 12 ]

Secondary Outcome Measures :
  1. Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
    Assessed by Modified Performance Oriented Mobility Assessment-Gait (mPOMA-G)

  2. Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
    Assessed by Bayley Scales of Infant and Toddler Development (BSID-III)

  3. Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
    Assessed by Pediatric Evaluation of Disability Inventory - Computer Adaptive Test (PEDI-CAT)

  4. Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
    Assessed by Sleep onset latency (SOL)

  5. Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
    Assessed by Total sleep time (TST)

  6. Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
    Assessed by Wake after sleep onset (WASO)

  7. Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
    Assessed by Nocturnal awakenings

  8. Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
    Assessed by Sedentary behavior

  9. Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
    Assessed by Daily activity level

  10. Exploratory efficacy measurement for behavior [ Time Frame: Change from baseline to week 12 ]
    Assessed by Aberrant Behavior Checklist (ABC)

  11. Exploratory efficacy measurement for behavior [ Time Frame: Change from baseline to week 12 ]
    Assessed by Anxiety, Depression, and Mood Scales (ADAMS)

  12. Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
    Assessed by EuroQol 5 Dimension 5 Level (EQ5D5L)

  13. Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
    Assessed by Child Health Assessment Questionnaire (CHAQ)

  14. Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
    Assessed by Parent Global Impression (PGI)

  15. Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
    Assessed by Short Form Health Survey 36 Items (SF-36)

  16. Exploratory efficacy measurement for overall clinical change [ Time Frame: Change from baseline to week 12 ]
    Assessed by Clinical Global Impression - Improvement (CGI-I)

  17. Exploratory efficacy measurement for overall clinical change [ Time Frame: Change from baseline to week 12 ]
    Assessed by Clinical Global Impression - Severity (CGI-S)


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   13 Years to 49 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  1. Age 13- 49 years
  2. Diagnosis of Angelman syndrome
  3. Receiving a stable regimen of concomitant medications for at least 4 weeks prior to Baseline, and able to maintain these throughout the duration of the study
  4. Has a caregiver capable of providing informed consent on behalf of the subject and able to attend scheduled study visits
  5. Able to ingest study medication
  6. Caregivers must agree not to post any subject or study information on social media

Exclusion Criteria

  1. Unable to perform the study related safety and exploratory efficacy assessments, such as motor function
  2. Poorly controlled seizure activity
  3. Concomitant cardiovascular, respiratory, liver, renal, or hematologic diseases of a degree that would limit participation in the study
  4. Pregnancy or women of child-bearing potential who are not using and acceptable method of contraception
  5. Concomitant use of minocycline, levodopa, zolpidem, zaleplon, eszopiclone, ramelteon, and cannabinoid derivatives, or any other use of any investigational agent, device, and/or investigational procedure 4 weeks prior to Baseline and during the study
  6. Allergy to OV101 or any excipients
  7. At increased risk of harming self and/or others based on investigator assessment
  8. Any condition or reason that in the opinion of the investigator makes the subject unsuitable for enrollment
  9. Inability of subject or caregiver to comply with study requirements

Other protocol-defined inclusion/exclusion criteria may apply.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02996305


Contacts
Contact: Amit Rakhit, MD, MBA +1 (646) 661-7661 info@ovidrx.com

Locations
United States, Arizona
Ovid Therapeutics Investigative Site Recruiting
Phoenix, Arizona, United States
United States, California
Ovid Therapeutics Investigative Site Recruiting
San Diego, California, United States
United States, Florida
Ovid Therapeutics Investigative Site Recruiting
Gainesville, Florida, United States
Ovid Therapeutics Investigative Site Recruiting
Tampa, Florida, United States
United States, Georgia
Ovid Therapeutics Investigative Site Recruiting
Atlanta, Georgia, United States
United States, Illinois
Ovid Therapeutics Investigative Site Recruiting
Chicago, Illinois, United States
United States, Massachusetts
Ovid Therapeutics Investigative Site Recruiting
Boston, Massachusetts, United States
Ovid Therapeutics Investigative Site Recruiting
Lexington, Massachusetts, United States
United States, Ohio
Ovid Therapeutics Investigative Site Recruiting
Cincinnati, Ohio, United States
United States, Pennsylvania
Ovid Therapeutics Investigative Site Recruiting
Media, Pennsylvania, United States
United States, South Carolina
Ovid Therapeutics Investigative Site Recruiting
Greenwood, South Carolina, United States
United States, Tennessee
Ovid Therapeutics Investigative Site Recruiting
Nashville, Tennessee, United States
Israel
Ovid Therapeutics Investigative Site Recruiting
Ramat Gan, Israel
Sponsors and Collaborators
Ovid Therapeutics Inc.
Investigators
Study Director: Amit Rakhit, MD, MBA Ovid Therapeutics Inc.
More Information

Responsible Party: Ovid Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT02996305     History of Changes
Other Study ID Numbers: OV101-15-001
First Posted: December 19, 2016    Key Record Dates
Last Update Posted: November 30, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Anticonvulsants
Syndrome
Angelman Syndrome
Disease
Pathologic Processes
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Gaboxadol
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
GABA Agonists
GABA Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action